Search All Tech Briefings


Search technology briefings produced by NIHR Innovation Observatory. These are short reports on new or repurposed medicines that we have already sent to our stakeholders. Only those technologies (medicines) that are within the scope of our stakeholders will have a technology briefing available.  Search our technology briefings by intervention (drug or device), specific indication (disease or condition), or general therapeutic area.

| 90 Results

Title
Intervention Indication Therapeutic Area Year Actions
Leniolisib for previously untreated activated phosphoinositide 3-kinase delta syndrome Leniolisib Activated PI3K delta syndrome Genetic Disorders 2022 View  |  Download
Lenadogene Nolparvovec (GS-010) for Vision Loss from Leber's Hereditary Optic Neuropathy Due to Mutation of the ND4 Gene Lenadogene nolparvovec (Lumevoq; GS-010) Leber's hereditary optic neuropathy (LHON) Genetic Disorders , Ophthalmology 2017 View  |  Download
Lanadelumab for prevention of attacks in adults and adolescents with Type I and Type II hereditary angioedema Lanadelumab (SHP643; TAK-743; Takhzyro) Hereditary angioedema (HAE) Genetic Disorders , Immunology 2017 View  |  Download
Kaftrio (Elexacaftor/tezacaftor/ivacaftor) and ivacaftor for treating cystic fibrosis in children (2 to 5 years) with at least one F508del mutation in the CFTR gene Kaftrio (fixed dose combination: Elexacaftor; Tezacaftor; Ivacaftor) Cystic fibrosis Genetic Disorders , Respiratory System 2022 View  |  Download
Ivacaftor for cystic fibrosis in infants Ivacaftor (Kalydeco; VX-770) Cystic fibrosis Genetic Disorders , Respiratory System 2023 View  |  Download
Inclisiran for primary hypercholesterolaemia and mixed dyslipidaemia Inclisiran (ALN-PCSsc; ALN-60212) Heterozygous familial hypercholesterolaemia (HeFH) , Primary hypercholesterolaemia Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2019 View  |  Download
Human C1-esterase inhibitor (subcutaneous injection) for preventing hereditary angioedema Human C1 esterase inhibitor (Berinert) Hereditary angioedema (HAE) Genetic Disorders , Immunology 2023 View  |  Download
Givinostat for Duchenne muscular dystrophy Givinostat Duchenne muscular dystrophy (DMD) Genetic Disorders , Musculoskeletal System 2023 View  |  Download
Garadacimab for preventing attacks of hereditary angioedema Garadacimab Hereditary angioedema (HAE) Genetic Disorders , Immunology 2023 View  |  Download
Fordadistrogene movaparvovec for Duchenne muscular dystrophy Fordadistrogene movaparvovec Duchenne muscular dystrophy (DMD) Genetic Disorders , Musculoskeletal System 2022 View  |  Download
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